Application of neuromuscular organoids for the development of personalized therapy in spinal muscular atrophy


Grant Data
Project Title
Application of neuromuscular organoids for the development of personalized therapy in spinal muscular atrophy
Principal Investigator
Professor Cheung, Martin Chi Hang   (Project Coordinator (PC))
Co-Investigator(s)
Dr Xiong Wenjun   (Co-principal investigator)
Professor Javed Asif   (Co-principal investigator)
Professor Chan Hoi Shan Sophelia   (Co-principal investigator)
Duration
36
Start Date
2024-06-01
Amount
5714286
Conference Title
Application of neuromuscular organoids for the development of personalized therapy in spinal muscular atrophy
Keywords
Neuromuscular organoids, Single cell RNA sequencing, Motor neurons, Disease modelling, Spinal muscular atrophy
Discipline
Disease Model DevelopmentPersonalized Medicine
Panel
Biology and Medicine (M)
HKU Project Code
R7018-23
Grant Type
Research Impact Fund (RIF) 2023/24
Funding Year
2023
Status
On-going
Objectives
(1) To generate patient-specific neuromuscular organoids (NMOs) for modeling spinal muscular atrophy (SMA) pathogenesis(2) To identify pathogenic factors and cell types responsible for MN and skeletal muscle degeneration by single-cell transcriptomics(3) To evaluate the therapeutic potential of candidate factors from single-cell transcriptomics in SMA NMOs and the SMA mouse model